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2-STEP: A single-centre, phase II study to evaluate the safety, tolerability and pharmacokinetics of 2-Iminobiotin (2-IB) in neonates with gestational age of *36 weeks with moderate to severe perinatal asphyxia treated with therapeutic hypothermia

To explore the safety, tolerability and the pharmacokinetic profile of 2-IB when given on top of therapeutic hypothermia.

Thyroid hormone analog therapy of patients with severe psychomotor retardation caused by mutations in the MCT8 thyroid hormone transporter: The Triac Trial.

1. Normalization of the abnormal serum TH parameters and thereby improving the clinical condition of the AHDS patients2. Observation of changes in cognitive and motor function.

An open label exploratory dose finding and pharmacokinetic clinical trial of bumetanide for the treatment of NEonatal seizure using Medication Off-patent (NEMO).

The aim of the trial is to obtain data on the optimal dose, feasibility and pharmacokinetics of bumetanide when given as an add-on treatment for seizures in full term babies with hypoxic ischemic encephalopathy (HIE). Bumetanide will be given in a…

Prevention of communicative decline in the cognitively impaired. Intervention on disordered perceptive and productive functions.

Aim of this project is to diagnose hearing impairment, and underlying disorders in speech perception and production, and to develop and implement adequate intervention techniques, which are subsequently evaluated. The ultimate goal is to prevent…

The effect of Targeted Training Therapy(TTT) on children with Cerebral Palsy

Objective is to study the effect of Targeted Training Therapy on balance control in children with Cerebral Palsy.

A Global, Multicenter, Single-arm, Matched External Control Study of Intrathecal SHP611 in Subjects with Late Infantile Metachromatic Leukodystrophy

The primary objective of this study is to evaluate the effects of intrathecal (IT) administration of SHP611 (also known as TAK-611) on the time to loss of locomotion, as indicated by category 5 or higher in the Gross Motor Function Classification in…

A Proof-of-Concept Study to Explore the Potential Efficacy of Deferiprone in Patients With
Pelizaeus-Merzbacher disease (PMD)

This study has been transitioned to CTIS with ID 2024-511968-81-00 check the CTIS register for the current data. Studying the effect of deferiprone on the motor function of patients with Pelizaeus-Merzbacher disease.

A randomised, double-blind, controlled trial to evaluate the effects of a nutritional product on brain integrity in preterm infants

>Primary objective:During the intervention period (from randomisation until and including Term Equivalent Age [TEA]): To investigate the effect of the test product vs. the control product given to preterm infants born at 24+0 to <30+0…